PharmMedProm: Aston Health Experts Discussed the Challenges and Outlook for Orphan Disease Treatment in Russia
Publication date: 2 february 2021
During the online business breakfast "Orphan Diseases in Russia: Ten Important Years" arranged by Aston Health, the company's experts presented their perspectives on challenges and opportunities related to treatment of orphan conditions in this country.
The discussion held prior to International Day of Rare (Orphan) Diseases (Feb. 29) brought together Anton Artyomov, CEO, Aston Health, and the heads of company divisions: Alexander Ivanov, Strategic Consulting, Yekaterina Lavrentieva, Medical Programs, Natalia Mueva, Laboratory Diagnostics, Elizaveta Karpikhina, Marketing Research and Andrey Kazakov, Orphan Diseases.
It was noted that since Federal Law # 323 “On the Fundamentals of Health Protection …” came into effect in 2011, introducing the definition of orphan conditions the "7 Nosologies" program was adopted that now covers such orphan conditions as hemophilia, cystic fibrosis, pituitary dwarfism and Gaucher disease. Also, the federal database of patients with these conditions was developed.
Aston Health has been involved in the marketing and epidemiological studies for PNH, Gaucher disease, MPS, Pompe disease and Fabry disease.
Also, since 2016, Aston Health in collaboration with the Medical Genetics Research Center n.a. Bochkov has been performing the Audit of Care Delivery for Patients with Orphan Diseases aimed at setting up a comprehensive patient registry. The current database includes information on over 5,000 patients presenting with various orphan diseases (20 conditions).
However, despite the government efforts and the steps taken by patient communities and pharmaceutical companies, orphan disease patients face a number of challenges and hurdles, including:
- Lack of appropriate awareness among clinicians;
- Challenges related to diagnosis, as well as timely diagnosis of orphan conditions and access to treatment;
- Lack of structured system for pharmaceutical provision of such patients.
According to Aston Health experts, to improve diagnosis and post-doagnostic support it is necessary to:
- Develop competences of healthcare providers;
- Raise awareness about the available diagnostic options not covered by OMS;
- Engage a larger number of certified testing labs.
Also, to improve the quality of care in this area, the following steps are required:
- Streamline the collection, processing and analysis of information on orphan disease patients;
- Improve the approaches to pharmaceutical provision.
Significant steps in this direction are already being made, including the creation of the Krug Dobra Foundation to support children with severe life-threatening and chronic diseases. The Russian Government made a decision to allocate 60 billion rubles for the purchase of medications (drugs registered in Russia) for these pediatric patients. Meanwhile, a lot of debate is unfolding regarding unregistered medications that the orphan disease patients need for treatment. Also, a new online platform for monitoring the spending of allocated funds will be launched soon as part of the government efforts to improve transparency and accountability.
The observations and inferences by Aston Health experts were supported by Olga Germanenko, founder and director of the SMA Charity Fund for Patients with SMA and Other Neuromuscular Disorders “SMA Families”. She noted that important steps are being taken now and expressed hope that Krug Dobra would soon be able to extend support to adult patients. The meeting participants stressed the importance of further integration with government agencies, patient communities and pharmaceutical companies to come up with effective solutions for orphan disease patients.
Text: Irina Gracheva
Source: PharmMedProm website